What is the role of ezetimibe in the treatment of sitosterolemia (phytosterolemia)?

Updated: May 24, 2019
  • Author: Robert D Steiner, MD; Chief Editor: Luis O Rohena, MD, MS, FAAP, FACMG  more...
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In October 2002, a new cholesterol absorption inhibitor, ezetimibe, received US Food and Drug Administration (FDA) approval for use in sitosterolemia. Because the mechanism by which it inhibits cholesterol absorption is quite specific, the adverse effects and drug interactions associated with the resins should not be expected.

Ezetimibe can increase platelet count and decrease mean platelet volume, thereby potentially reducing the risk for bleeding in sitosterolemia. [25]

A multiple center collaborative randomized placebo-controlled study of ezetimibe 10 mg/d in patients aged 10 years and older determined that ezetimibe was well tolerated and efficacious in reducing plant sterol levels compared with a placebo. [26] Plasma cholesterol levels, if elevated, fall dramatically in patients treated with ezetimibe. Plasma plant sterol levels fall also but often not to normal levels.

A study in China documented the successful use of ezetimibe in several young children, although a child younger than 2 years did not initially respond. [5] The long-term efficacy and safety of ezetimibe in sitosterolemia have been documented. [27] In contrast with patients with adult sitosterolemia who typically reached full treatment response within 2–8 weeks of treatment, [26] 4 months of treatment was required to significantly lower the sterols in the children reported in the study in China.

Little data on the use of ezetimibe in children younger than 10 years are available. Information on the use of medications other than cholestyramine and ezetimibe in sitosterolemia is limited. Recently, administration of ezetimibe added to cholestyramine was reported in a patient with sitosterolemia with remarkably positive results. [28]

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